Guide on Methodological Standards in Pharmacoepidemiology in China (2nd edition) and their series interpretation (5): classic study designs and derivative approaches

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Author: ZHANG Yiying ¹ YIN Shiwenqian ¹ MENG Shuhan ² WANG Shanjie ³  ZHAN Siyan ^4, ^5, ^6, ⁷  SUN Feng ^4, ^5, ^7, ^8, ^9, ¹⁰

Affiliation: 1. Department of Epidemiology and Health Statistics, School of Public Health, Jiamusi University, Jiamusi 154007, Heilongjiang Province, China 2. Department of Epidemiology, School of Public Health, Harbin Medical University, Harbin 154000, China 3. Department of Cardiology, Second Affiliated Hospital of Harbin Medical University, Harbin 154000, China 4. Department of Epidemiology and Biostatistics, School of Public Health, Peking University, Beijing 100191, China 5. Key Laboratory of Epidemiology of Major Diseases (Peking University), Ministry of Education, Beijing 100191, China 6. Clinical Epidemiology Research Center, Peking University Third Hospital, Beijing 100191, China 7. Center for Post-Marketing Safety Evaluation of Drugs, Peking University Health Science Center, Beijing 100191, China 8. Department of Ophthalmology, Peking University Third Hospital, Beijing 100191, China 9. College of Traditional Chinese Medicine, Xinjiang Medical University, Urumqi 830017, China 10. School of Medicine, Shihezi University, Shihezi 832000, Xinjiang Uygur Autonomous Region, China

Keywords: Pharmacoepidemiology Methodology Guidelines Classic research type Derivative design

DOI: 10.12173/j.issn.1005-0698.202504163

Reference: ZHANG Yiying, YIN Shiwenqian, MENG Shuhan, WANG Shanjie, ZHAN Siyan, SUN Feng. Guide on Methodological Standards in Pharmacoepidemiology in China (2nd edition) and their series interpretation (5): classic study designs and derivative approaches[J]. Yaowu Liuxingbingxue Zazhi, 2025, 34(5): 485-493. DOI: 10.12173/j.issn.1005-0698.202504163.[Article in Chinese]  Copied

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Abstract

harmacoepidemiology is an interdisciplinary field that applies epidemiological methods to study drug use, effectiveness, and associated risk in populations. Standardizing research methods in this field is crucial for ensuring research quality and promote the development of the discipline. Based on the Guide on Methodological Standards in Pharmacoepidemiology in China(2nd edition), this article interprets the relevant contents about classic research types and their derivative designs. It aims to clarify the types of study methodological designs in pharmacoepidemiology, systematically describe the characteristics and applications classical derivative designs, and compare these with research design frameworks outlined in international pharmacoepidemiological guidelines. Compared to the first edition, the second edition of the guideline has updated and detailed the types of research, updating the research design to original research (interventional research and non-interventional research), secondary research (systematic review, Meta-analysis, economic analysis, etc.), and tertiary research (umbrella review, etc.). Additionally, a variety of derivative designs have been added, including target trial emulation, nested case-control and case-cohort studies, case-crossover designs, self-controlled case series and self-controlled risk interval designs, case-population studies, interrupted time-series analysis, and case-coverage (ecological) designs for vaccine surveillance. This article strengthens the operability of the theoretical guidance of pharmacoepidemiological research methods in practice and provides a reference for conducting high-quality pharmacoepidemiological research.

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References

1.Lawson DH. Pharmacoepidemiology: a new discipline[J]. BMJ, 1984, 289(6450): 940-941. DOI: 10.1136/bmj.289.6450.940.

2.Montastruc JL, Benevent J, Montastruc F, et al. What is pharmacoepidemiology? Definition, methods, interest and clinical applications[J]. Therapie, 2019, 74(2): 169-174. DOI: 10.1016/j.therap.2018.08.001.

3.胥洋, 丁呈怡, 詹思延, 等. 中国药物流行病学研究方法学指南[J]. 药物流行病学杂志, 2019, 28(1): 5-9. [Xu Y, Ding CY, Zhan SY, et al. Guide on Methodological Standards in Pharmacoepidemiology[J]. Chinese Journal of Pharmacoepidemiology, 2019, 28(1): 5-9.] DOI: 10.19960/j.cnki.issn1005-0698.2019.01.002.

4.中国药学会药物流行病学专业委员会. 中国药物流行病学研究方法学指南（T/CPHARMA 002-2019）[J]. 中华流行病学杂志, 2019, 40(10): 1180-1185. DOI: 10.3760/cma.j.issn.0254- 6450.2019.10.002.

5.颜济南, 吴昀效, 聂晓璐, 等. 《中国药物流行病学研究方法学指南（第2版）》的制订/修订过程[J]. 药物流行病学杂志, 2025, 34(2): 121-135. [Yan JN, Wu YX, Nie XL, et al. Revision process of the Guide on Methodological Standards in Pharmacoepidemiology (2nd edition)[J]. Chinese Journal of Pharmacoepidemiology, 2025, 34(2): 121-135.] DOI: 10.12173/j.issn.1005-0698.202502028.

6.吴昀效, 颜济南, 聂晓璐, 等. 《中国药物流行病学研究方法学指南（第2版）》及其系列解读（1）: 概述[J]. 药物流行病学杂志, 2025, 34(1): 2-11. [Wu YX, Yan JN, Nie XL, et al. Guide on Methodological Standards in Pharmacoepidemiology in China (2nd edition) and their series interpretation (1): an overview[J]. Chinese Journal of Pharmacoepidemiology, 2025, 34(1): 2-11.] DOI: 10.12173/j.issn.1004-5511.202412131.

7.Sabaté M, Montané E. Pharmacoepidemiology: an overview[J]. J Clin Med, 2023, 12(22): 7033. DOI: 10.3390/jcm12227033.

8.Zhang L, Wong LY, He Y, et al. Pharmacovigilance in China: evolution and future directions[J]. Drug Saf, 2014, 37(10): 765-770. DOI: 10.1007/s40264-014-0222-3.

9.程吟楚, 何娜, 闫盈盈, 等. 《中国药物流行病学研究方法学指南（第2版）》及其系列解读（4）: 药物流行病学研究的问题来源与方案制定[J]. 药物流行病学杂志, 2025, 34(4): 365-372. [Cheng YC, He N, Yan YY, et al. Guide on Methodological Standards in Pharmacoepidemiology in China (2nd edition) and their series interpretation (4): sources of research question and study protocol development in pharmacoepidemiological research[J]. Chinese Journal of Pharmacoepidemiology, 2025, 34(4): 365-372.] DOI: 10.12173/j.issn.1005-0698.202504010.

10.Caparrotta TM, Dear JW, Colhoun HM, et al. Pharmacoepidemiology: using randomised control trials and observational studies in clinical decision-making[J]. Br J Clin Pharmacol, 2019, 85(9): 1907-1924. DOI: 10.1111/bcp.14024.

11.王俊彦, 程吟楚, 周鹏翔, 等. 药物流行病学研究的设计原理与方法简介[J]. 药物流行病学杂志, 2021, 30(5): 291-295. [Wang JY, Cheng YC, Zhou PX, et al. An introduction to the design principles and methods of pharmacological epidemiological research[J].Chinese Journal of Pharmacoepidemiology, 2021, 30(5): 291-295.] DOI: 10.19960/j.cnki.issn1005-0698.2021.05.001.

12.Hernán MA, Wang W, Leaf DE. Target trial emulation: a framework for causal inference from observational data[J]. JAMA, 2022, 328(24): 2446-2447. DOI: 10.1001/jama.2022.21383.

13.江流, 谢亮亮, 严双琴, 等. 生命早期抗生素暴露对6~11月龄和18~23月龄儿童过敏性疾病影响的出生队列研究[J]. 中华预防医学杂志, 2021, 55(5): 598-605. [Jiang L, Xie LL, Yan SQ, et al. Birth cohort study on the impact of early antibiotic exposure on allergic diseases in children aged 6-11 months and 18-23 months[J]. Chinese Journal of Preventive Medicine, 2021, 55(5): 598-605.] DOI: 10.3760/cma.j.cn112150-20210316-00257.

14.Tenny S, Kerndt CC, Hoffman MR. Case control studies[M]. Treasure Island (FL): StatPearls Publishing, 2023.

15.王巍巍, 卓琳, 詹思延, 等. 药械安全性评价研究的系统综述和Meta分析制作指南[J]. 药物流行病学杂志, 2020, 29(7): 502-509. [Wang WW, Zhuo L, Zhan SY, et al. Systematic review and Meta-analysis of drug and medical device safety evaluation research[J].Chinese Journal of Pharmacoepidemiology, 2020, 29(7): 502-509.] DOI: 10.19960/j.cnki.issn1005-0698.2020.07.011.

16.Li D, Wang Y. Safety of azithromycin in pediatric infectious diseases: a clinical systematic review and Meta-analysis[J]. Transl Pediatr, 2021, 10(10):2594-2601. DOI: 10.21037/tp-21-444.

17.Petimar J, Young JG, Yu H, et al. Medication-induced weight change across common antidepressant treatments: a target trial emulation study[J]. Ann Intern Med, 2024, 177(8): 993-1003. DOI: 10.7326/M23-2742.

18.Pottegård A. Core concepts in pharmacoepidemiology: fundamentals of the cohort and case-control study designs[J]. Pharmacoepidemiol Drug Saf, 2022, 31(8): 817-826. DOI: 10.1002/pds.5482.

19.Daneman N, Cheng Y, Gomes T, et al. Metronidazole-associated neurologic events: a nested case-control study[J]. Clin Infect Dis, 2021, 72(12): 2095-2100. DOI: 10.1093/cid/ciaa395.

20.Poulsen AH, Sears CG, Harrington J, et al. Urinary cadmium and stroke-a case-cohort study in Danish never-smokers[J]. Environ Res, 2021, 200: 111394. DOI: 10.1016/j.envres.2021.111394.

21.Bots SH, Brown J, Wong AYS, et al. Core concepts: self-controlled designs in pharmacoepidemiology[J]. Pharmacoepidemiol Drug Saf, 2025, 34(1): e70071. DOI: 10.1002/pds.70071.

22.Man KKC, Lau WCY, Coghill D, et al. Association between methylphenidate treatment and risk of seizure: a population-based, self-controlled case-series study[J]. Lancet Child Adolesc Health, 2020, 4(6): 435-443. DOI: 10.1016/S2352-4642(20)30100-0.

23.Hallas J, Pottegård A. Use of self-controlled designs in pharmacoepidemiology[J].J Intern Med, 2014, 275(6): 581-589. DOI: 10.1111/joim.12186.

24.Liu CH, Yeh YC, Huang WT, et al. Assessment of pre-specified adverse events following varicella vaccine: a population-based self-controlled risk interval study[J]. Vaccine, 2020, 38(11): 2495-2502. DOI: 10.1016/j.vaccine.2020.01.090.

25.Eworuke E, Welch EC, Tobenkin A, et al. Use of FDA's Sentinel System to quantify seizure risk immediately following new ranolazine exposure[J]. Drug Saf, 2019, 42(7): 897-906. DOI: 10.1007/s40264-019-00798-2.

26.于石成, 王琦琦, 毛凡, 等. 中断时间序列设计及其分析方法 [J]. 中华预防医学杂志, 2019, 53(8): 858-864. [Yu SC, Wang QQ, Mao F, et al. Interrupted time series design and its analysis methods[J]. Chinese Journal of Preventive Medicine, 2019, 53(8): 858-864.] DOI: 10.3760/cma.j.issn.0253-9624.2019.08.012.

27.颜济南, 吴昀效, 聂晓璐, 等. 《中国药物流行病学研究方法学指南（第2版）》系列解读（3）: 全球指南的对比[J]. 药物流行病学杂志, 2025, 34(3): 241-259 .[Yan JN, Wu YX, Nie XL, et al. Interpretation series of the Guide on Methodological Standards in Pharmacoepidemiology in China (2nd edition) (3): comparison of global guidelines[J]. Chinese Journal of Pharmacoepidemiology, 2025, 34(3): 241-259.] DOI: 10.12173/j.issn.1005-0698.202503119.

28.The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). Guide on Methodological Standards in Pharmacoepidemiology (Revision 11) [R/OL]. (2024-05-04) [2025-04-25]. https://encepp.europa.eu/encepp-toolkit/methodological-guide_en.

29.Public Policy Committee, International Society of Pharmacoepidemiology. Guidelines for good pharmacoepidemiology practice (GPP)[J]. Pharmacoepidemiol Drug Saf, 2016, 25(1): 2-10. DOI: 10.1002/pds.3891.

30.European Medicines Agency. Guideline on good pharmacovigilance practices (GVP) Module VIII-Post-authorisation safety studies (rev 3) [S/OL]. (2024-05-04) [2025-04-25]. https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-good-pharmacovigilance-practices-gvp-module-viii-post-authorisation-safety-studies-rev-3_en.pdf.